Much of the research into a cure for retinitis pigmentosa is genotype specific. When Usher 1F Collaborative was founded in December 2013, there was minimal research for type 1F. Today, we have provided over $1 million in funding to four major research labs that specialize in Usher Syndrome research. At two of these labs, researchers have created animal models, a zebrafish and a mouse, that exhibit deafness, poor balance, and progressive vision loss. They are now using these animal models to develop and test gene and drug therapies. Another lab has achieved major progress toward achieving both gene replacement therapy and stem cell therapy using retinal cells made from patients’ own skin cells. With continued funding, these paths to a cure have the potential to result in a clinical trial in single digit years.
Right now, parents of children with type 1F are sitting and watching their children's vision fade while clinical trials for other types of Usher Syndrome are offering encouragement and hope to others. Please help make hope a reality for those with Usher 1F.